Frontera Therapeutics has dosed the first participant in a Phase I clinical trial of FT-001 to treat Leber Congenital Amaurosis-2 (LCA-2), a severe inherited retinal disease that is related to a mutation in the RPE65 gene.
The Phase I clinical trial has been designed for assessing FT-001’s pharmacokinetics, therapeutic effects, tolerability, and safety as a single dose in LCA-2 patients.
It is being conducted in China and the initial data is anticipated in the second half of this year.
The China National Medical Products Administration’s (NMPA) Center for Drug Evaluation (CDE) accepted the company’s investigational new drug (IND) application for FT-001 in September last year.
In April, the company obtained IND clearance for FT-001 from the US Food and Drug Administration (FDA).
Frontera Therapeutics CEO and founder Yong Dai said: “This is an exciting achievement for Frontera Therapeutics as we begin our first clinical trial and take a significant step forward as a company.”
The company’s lead AAV gene therapy product candidate, FT-001, is given through a one-time injection into the subretinal space of the eye.
It provides a functional copy of the human RPE65 gene to the nuclei of the retinal cells of the patient.
The addition of a functional copy of the RPE65 gene enables the expression of normal functioning human hRPE65 proteins, which improve the functional vision of a patient.
According to the company, LCA is classified as an ultra-orphan disease and is said to be found in two to three out of every 100,000 new borns.
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